Safety of Ruconest in children with HAE

Phase 1

• Conditions: Attacks associated with hereditary angioedema (HAE) in pediatric population 2-13 years of age.; MedDRA version: 18.1Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2011-000987-92-CZ
• Lead Sponsor: Pharming Technologies B.V.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-04-17
• Study Completion: 2017-07-17
• Last Update Posted: 4 January 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Screening
•From 2 up to and including 13 years of age
•Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of normal)
•Signed written informed consent (parental permission) signed by the legal guardian(s)

Treatment
•Clinical symptoms of an acute HAE attack
•Onset of eligible symptoms within 5 hours from the moment at which medical evaluation to determine eligibility has occurred
•IS score for at least one anatomical location at the time of initial evaluation of at least 3 (moderate severity or greater) without signs of spontaneous regression
•24h or more have passed since the patient’s last study treatment

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Screening
•A diagnosis of acquired C1INH deficiency (AAE)
•A medical history of allergy to rabbits or rabbit-derived products (including rhC1INH, antisera), or positive anti-rabbit epithelium (dander) IgE test (cut off>0.35 kU/L in ImmunoCap® assay (Phadia, Sweden) or equivalent)
•Treatment with investigational drug in another clinical study in the last 30 days
•Any clinical significant abnormality in the physical examination and/or the routine laboratory assessments, that in the opinion of the Investigator makes the patient unsuitable for participation in the study
•Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives

Treatment
•Any changes since screening that would exclude patient based on above exclusion criteria.
•10 HAE attacks were previously treated with study medication.
•Suspicion for an alternate explanation of the symptoms other than an acute HAE attack.
•Use of any disallowed concomitant medication since onset of acute HAE attack

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the clinical safety, immunogenicity and tolerability of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.;Secondary Objective: To assess the pharmacokinetics (PK) and pharmacodynamics (PD) of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.<br><br>To assess the efficacy of Ruconest in the treatment of acute angioedema attacks in 2-13 year old HAE patients.<br>;Primary end point(s): Assessment of safety and tolerability.;Timepoint(s) of evaluation of this end point: This will be evaluated by continuous recording adverse events.<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Time to beginning of relief, time to minimal symptoms, time to complete resolution.<br>Pharmacokinetic and pharmacodynamic parameters (C1INH activity and C4 in plasma) during treatment for the first attack.;Timepoint(s) of evaluation of this end point: The clinical endpoints will be evaluated following the treatment of an attack. Blood samples for PK and PD will be taken at 5 minutes and between 2-4 hours after the Ruconest injection at the first attack.